A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

Inclusion Criteria

• •Type of Participant and Disease Characteristics:
• •Clinical diagnosis of CPVT, based on documented history of polymorphic or bidirectional non-sustained ventricular tachycardia with exercise or ventricular ectopy in a pattern consistent with CPVT on EST.
• •Central Screening laboratory determination of a RYR2 variant that is pathogenic or likely pathogenic for CPVT.
• •Documented history of life-threatening ventricular arrhythmic event defined as: survived sudden cardiac arrest, sudden cardiac arrest with appropriate implantable cardioverter defibrillator (ICD) shock, arrhythmic syncope, or sustained ventricular tachycardia (30 seconds or more) with or without ICD shock.
• •On stable dose (defined as no change in dose by more than 50% for at least 1 month prior to Screening) of standard-of-care therapy defined as a beta-blocker and/or flecainide.
• •Documented prior history of EST demonstrating a ventricular arrythmia score (VAS) score of ≥ 2.
• •For the first 2 participants in each cohort only: a properly functioning ICD device in place. Following review of data from Cohorts 1 and 2, the Data Safety and Monitoring Board (DSMB) will determine if this criterion is required for participants in Cohort 3.
• •Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this.